The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

Roche’s subsidiary Genentech has shared positive Phase III data with the US Food and Drug Administration (FDA) as the company targets the billion-dollar lupus market for its CD20-targeting drug Gazyva ...

UMD researchers have discovered key mechanisms in gene regulation that could improve the design of RNA-based medicines.

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

As part of The Wistar Institute's Melanoma Research Center, the Villanueva lab pursues new ways to overcome the challenge of melanoma drug resistance. In this paper, the group took aim at melanomas ...

United Therapeutics has scored a regulatory green light to start clinical trials for its genetically modified pig kidneys to ...

Wistar Institute scientists identified a new strategy for attacking treatment-resistant melanoma: inhibiting the gene S6K2.

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

In this manuscript, we give an overview of historical aspects, as well as the current state of clinical trials and approved gene therapy products for commercialization in Brazil. Focusing on gene ...